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ARBM-301

TARGET INDICATION DEVELOPMENT STAGE
Pendrin Pendred Syndrome Qualified Leads to Preclinical Candidate Selection

ARBM-301

TARGET

Pendrin

INDICATION

Pendred Syndrome

DEVELOPMENT STAGE

Qualified leads to Preclinical Candidate Selection

Summary

Pendred syndrome is inherited in an autosomal recessive manner by genetic mutations on SLC26A4. The structural abnormality caused by genetic mutations on SLC26A4 prevents pendrin protein expression on the cell surface, which results in pH disturbance within the inner ear, leading to hearing loss. The structure of pendrin protein corrected by ARBM-301 makes its surface expression restored and its function also rescued as an anion exchanger.

Mechanism of Action

ARBM-301 is designed to rescue pendrin protein from degradation by correcting its conformation of the mutated pendrin protein retained in the ER thereby allowing its targeting to the cell surface with further post-translational modifications in the TGN on the way.

Efficacy

The rescuing effect of pendrin correctors on the disabled hearing of the patients with SLC26A4 mutations.